A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa.

miglustat - Generic mfg.

Pombiliti (cipaglucosidase alfa-atga) - BioMarin

Myozyme (alglucosidase alfa) - Sanofi

http://www.ncbi.nlm.nih.gov/pubmed/42454581

Alasdair MacCulloch; Amy Dymond; Jordi Díaz-Manera; Matthew Crabtree; Neil Johnson; Patrick Deegan; Simon Shohet; Sophie Clarke; Vera Gielen; Will Green

Jul 16, 2026